BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial was a single-center, double-blind, placebo-controlled, single-dose escalation study followed by a multiple-dose escalation study in healthy volunteers.
The administration of up to 400 mg/kg did not achieve plasma concentrations believed to be required to increase utrophin expression. Moreover, plasma concentrations of BMN 195 were even lower on repeat administration. Based on these results, BioMarin has concluded that the likelihood of achieving a therapeutic effect in DMD patients is highly unlikely and has discontinued development of BMN 195.
"Duchenne muscular dystrophy remains a serious unmet medical need affecting approximately 40,000 patients in the developed world, and BioMarin remains committed to this disease area," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Given the limitations of BMN 195, we believe that other approaches to up-regulation of utrophin may be more possible, and we continue to believe that utrophin upregulation is a viable approach for the treatment of DMD. We are currently working on additional candidates to take forward into early human studies, and the new compound we are working on appears to overcome the limitations of BMN 195."
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